On November 4, 2026 Tango Therapeutics, Inc. (NASDAQ: TNGX), a clinical-stage biotechnology company committed to delivering breakthrough targeted therapies that extend and improve the lives of patients with cancer and Erasca, Inc. (Nasdaq: ERAS), a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK pathway-driven cancers, reported that the Phase 1/2 study of oral PRMT5 inhibitor vopimetostat in combination with pan-RAS molecular glue ERAS-0015 in patients with MTAP-deleted pancreatic ductal adenocarcinoma (PDAC) and MTAP-deleted/RAS-mutant non-small cell lung cancer (NSCLC) is now open for enrollment.

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"The encouraging clinical activity, durability and tolerability observed to date with vopimetostat in combination with RAS inhibition reinforce our belief that co-targeting PRMT5 and RAS represents a promising new frontier in the treatment of pancreatic cancer and potentially other RAS-driven cancers," said Malte Peters, M.D., President and CEO of Tango Therapeutics. "We are thrilled to initiate this study in partnership with Erasca, a company leading the advancement of RAS-targeted therapies, to evaluate vopimetostat with ERAS-0015 and further explore the potential of this clinically validated and synergistic combination approach."

Jonathan E. Lim, M.D., Erasca’s chairman, CEO, and co-founder added, "Pairing ERAS-0015’s pan-RAS inhibition with vopimetostat’s PRMT5 inhibition creates a complementary, dual-targeted approach with the potential for deeper, more durable responses in patients with these difficult-to-treat cancers. We are excited to work closely with Tango, a leader in the development of PRMT5-targeted therapies, to advance this promising combination and evaluate its potential to deliver meaningful benefit for patients with pancreatic and lung cancers, where new treatment options are urgently needed."

About the Phase 1/2 Study of Vopimetostat in Combination with ERAS-0015
The Phase 1/2 open-label, multicenter dose-escalation and expansion trial, sponsored by Tango Therapeutics, is designed to evaluate vopimetostat in combination with ERAS-0015 in patients with MTAP-deleted PDAC or RAS-mutant/MTAP-deleted NSCLC. The primary objective of the dose-escalation portion of the study is to determine the maximum tolerated dose of the combination regimen. Secondary objectives include assessment of antitumor activity, pharmacokinetics, safety and tolerability. To learn more about this study, visit clinicaltrials.gov (NCT07897734).

About Vopimetostat
Vopimetostat is an investigational, potentially first- and best-in-class oral, once-daily, PRMT5 inhibitor that is being evaluated as a monotherapy and as a combination therapy in multiple indications. In ongoing clinical studies, vopimetostat has demonstrated a favorable safety and tolerability profile and shown durable activity in multiple tumor types with MTAP (methylthioadenosine phosphorylase) deletion, including pancreatic cancer and lung cancer.

About ERAS-0015
ERAS-0015 is an oral, highly potent pan-RAS molecular glue designed to inhibit RAS signaling with a potential best-in-class profile. Erasca is evaluating ERAS-0015 in the AURORAS-1 Phase 1 trial in patients with RAS-mutant solid tumors. Early dose escalation data in AURORAS-1 demonstrated favorable safety and tolerability, well-behaved, linear PK, and confirmed and unconfirmed responses in multiple patients across multiple tumor types with different RAS mutations, including confirmed and unconfirmed partial responses at doses as low as 8 mg once daily (QD). In preclinical studies versus RMC-6236, ERAS-0015 demonstrated approximately 8-21 times higher binding affinity to cyclophilin A (CypA), approximately 5 times greater potency in RAS inhibition, and greater in vivo antitumor activity in RAS-driven tumor models evidenced by achieving comparable or greater tumor growth inhibition or regression at doses that are as low as approximately one-tenth to one-fifth of the dose of RMC-6236. ERAS-0015 is also designed to prevent resistance against mutant-selective inhibitors through inhibition of RAS wildtype variants. In addition, ERAS-0015 has demonstrated favorable absorption, distribution, metabolism, and excretion (ADME) and pharmacokinetic (PK) properties in multiple animal species.

(Press release, Tango Therapeutics, NOV 4, 2026, View Source [SID1234674244])

On November 4, 2026 Secura Bio, Inc. (www.securabio.com), an integrated pharmaceutical company maximizing commercial outcomes for oncology medicines, reported two posters on duvelisib, the Company’s oral dual inhibitor of PI3K-δ and PI3K-γ, have been accepted for presentation at the upcoming 2026 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place December 12-15, 2026, in New Orleans, Louisiana.

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A poster characterizing outcomes for peripheral T-cell lymphoma (PTCL) patients from the completed Phase 2 PRIMO study who achieved sufficient disease control with duvelisib to subsequently undergo stem cell transplant will be presented. Final results from the PRIMO study were published earlier this year in the Journal of Clinical Oncology.

A "Trials in Progress" poster detailing a new Phase 1 study for a novel topical formulation of duvelisib also will be presented. This is the first-ever clinical study evaluating a topical PI3K-δ/γ inhibitor in patients with early-stage Mycosis fungoides (MF), the most common subtype of cutaneous T-cell lymphoma (CTCL).

Presentation Details

Poster Number: 1834
Title: Duvelisib in Patients with Relapsed/Refractory Peripheral T-cell Lymphoma: Final Results from the Phase 2 PRIMO Trial: Outcomes in Patients who Proceeded to Stem Cell Transplant
Session Date: Saturday, December 12, 2026
Time: 5:30 pm – 6:30 pm EST
Location: MCCNO – Hall F
Presenter: Neha Mehta-Shah, M.D., Washington University School of Medicine, St. Louis, MO

Poster Number: 3619
Title: A Phase 1 dose-escalation study of topical duvelisib in patients with early-stage mycosis fungoides (MF)
Session Date: Sunday, December 13, 2026
Time: 6:00 pm – 8:00 pm EST
Location: MCCNO – Hall F
Presenter: Sima Rozati, M.D., Ph.D., Johns Hopkins University School of Medicine, Baltimore, MD

About Peripheral T-cell Lymphoma

Peripheral T-cell lymphoma (PTCL) is a rare, aggressive type of non-Hodgkin lymphoma that develops in mature white blood cells that circulate through the bloodstream and lymphatic system. PTCL accounts for between 10%-15% of all non-Hodgkin lymphomas and generally affects people aged 60 years and older. Although there are many different subtypes of PTCL, they often present in a similar way, with widespread, enlarged, typically painless lymph nodes in the neck, armpit, and/or groin. There are currently no well-established standards of care for patients with relapsed or refractory disease. ¹,²

About Cutaneous T-cell Lymphoma

Cutaneous T-cell lymphoma (CTCL) is a rare type of non-Hodgkin lymphoma that develops when white blood cells become abnormal and accumulate on the skin, presenting as painful, scaly patches, plaques or tumors that can spread across the body. Mycosis fungoides (MF) is the most common form of CTCL, affecting approximately 50%-60% of CTCL patients. Current treatment is generally broad-acting and not aimed at underlying biological causes of the disease.

About COPIKTRA (duvelisib)

COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K) and the first US approved dual inhibitor of PI3K-delta and PI3K-gamma pathways, two enzymes known to help support the growth and survival of malignant cells. PI3K signaling may lead to the proliferation of malignant cells and is thought to play a role in the formation and maintenance of a supportive tumor microenvironment. COPIKTRA is indicated in the United States for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior lines of systemic therapy.³ COPIKTRA is also being developed for the treatment of peripheral T-cell lymphoma (PTCL), for which it has received Fast Track status in the United States and is being investigated in combination with other agents through investigator-sponsored studies. It is also being developed for the treatment of cutaneous t-cell lymphoma (CTCL). Treatment of T-cell lymphomas is a disease category for which COPIKTRA is not currently indicated. For more information on COPIKTRA, please visit www.COPIKTRA.com.

INDICATIONS AND USAGE

COPIKTRA (duvelisib) is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior lines of systemic therapy.

Limitations of Use: COPIKTRA is not indicated or recommended for the treatment of any patients with CLL or SLL as initial or second line treatment due to an increased risk of treatment-related mortality.

IMPORTANT SAFETY INFORMATION

Treatment-related mortality occurred in 15% of COPIKTRA treated patients.
Fatal and/or serious infections occurred in 31% (4% fatal) of COPIKTRA-treated patients. Monitor for signs and symptoms of infection. Withhold COPIKTRA if infection is suspected.
Fatal and/or serious diarrhea or colitis occurred in 18% (<1% fatal) of COPIKTRA-treated patients. Monitor for the development of severe diarrhea or colitis. Withhold COPIKTRA.
Fatal and/or serious cutaneous reactions occurred in 5% (<1% fatal) of COPIKTRA-treated patients. Withhold COPIKTRA.
Fatal and/or serious pneumonitis occurred in 5% (<1% fatal) of COPIKTRA-treated patients. Monitor for pulmonary symptoms and interstitial infiltrates. Withhold COPIKTRA.
WARNINGS AND PRECAUTIONS

Treatment-related Mortality: In a randomized controlled trial in patients with relapsed or refractory CLL or SLL, treatment with COPIKTRA caused increased treatment-related mortality. With extended follow-up with a median of 63 months, treatment-related deaths occurred in 15% (23/158) of those patients in the overall population. In the indicated patient population, patients with relapsed or refractory CLL or SLL after at least two prior lines of systemic therapy. Treatment related deaths following treatment with COPIKTRA occurred in 14% (13/93) of patients. The most common cause of the treatment-related deaths were infections, which occurred in 9% and 11% of patients with relapsed or refractory CLL following at least one or two prior systemic therapies, respectively. COPIKTRA is not indicated and is not recommended for any patients in the initial or second-line treatment setting.

Infections: Serious, including fatal (18/442, 4%), infections occurred in 31% of patients receiving COPIKTRA. The most common serious infections were pneumonia, sepsis, and lower respiratory infections. Median time to onset of any grade infection was 3 months, with 75% of cases occurring within 6 months. Treat infections prior to initiation of COPIKTRA. Advise patients to report new or worsening signs and symptoms of infection. Cases of Pneumocystis jirovecii pneumonia (PJP) (1%) and cytomegalovirus (CMV) reactivation/infection (1%) occurred in patients taking COPIKTRA. Provide prophylaxis for PJP during treatment and following completion of treatment until the absolute CD4+ T cell count is greater than 200 cells/μL. Consider prophylactic antivirals during COPIKTRA treatment to prevent CMV infection, including CMV reactivation.

Diarrhea or Colitis: Serious, including fatal (1/442; 0.2%); diarrhea or colitis occurred in 18% of patients receiving COPIKTRA. Median time to onset of any grade diarrhea or colitis was 4 months, with 75% of cases occurring by 8 months. The median event duration was 0.5 months. Advise patients to report any new or worsening diarrhea.

Cutaneous Reactions: Serious, including fatal (2/442; 0.5%); cutaneous reactions occurred in 5% of patients receiving COPIKTRA. Fatal cases included drug reactions with eosinophilia and systemic symptoms (DRESS) and toxic epidermal necrolysis (TEN). Median time to onset of any grade cutaneous reaction was 3 months with a median event duration of 1 month. Presenting features for serious events were primarily described as pruritic, erythematous, or maculo-papular. Less common presenting features include exanthem, desquamation, erythroderma, skin exfoliation, keratinocyte necrosis, and papular rash. Advise patients to report new or worsening cutaneous reactions.

Pneumonitis: Serious, including fatal (1/442; 0.2%), pneumonitis without an apparent infectious cause occurred in 5% of patients receiving COPIKTRA. Median time to onset of any grade pneumonitis was 4 months with 75% of cases occurring within 9 months. The median event duration was 1 month with 75% of cases resolving by 2 months.

Hepatotoxicity: Grade 3 and 4 ALT and/or AST elevation developed in 8% and 2%, respectively, of patients receiving COPIKTRA (N=442). Two percent of patients had both an ALT or AST > 3 X ULN and total bilirubin > 2 X ULN. Median time to onset of any grade transaminase elevation was 2 months with a median event duration of 1 month. Monitor hepatic function during treatment with COPIKTRA.

Neutropenia: Grade 3 or 4 neutropenia occurred in 42% of patients receiving COPIKTRA (N=442), with Grade 4 neutropenia occurring in 24% of all patients. Median time to onset of grade ≥3 neutropenia was 2 months. Monitor neutrophil counts at least every 2 weeks for the first 2 months of COPIKTRA therapy, and at least weekly in patients with neutrophil counts < 1.0 Gi/L (Grade 3-4).

Embryo-Fetal Toxicity: COPIKTRA can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus and conduct pregnancy testing before initiating COPIKTRA treatment. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment and for 1 month after the last dose.

ADVERSE REACTIONS

B-cell Malignancies Summary

Fatal adverse reactions within 30 days of the last dose occurred in 8% (36/442) of patients treated with COPIKTRA 25 mg BID. Serious adverse reactions were reported in 289 patients (65%). The most frequent serious adverse reactions that occurred were infection (31%), diarrhea or colitis (18%), pneumonia (17%), rash (5%), and pneumonitis (5%). The most common adverse reactions (reported in ≥20% of patients) were diarrhea or colitis, neutropenia, rash, fatigue, pyrexia, cough, nausea, upper respiratory infection, pneumonia, musculoskeletal pain, and anemia.

CLL/SLL

Fatal adverse reactions within 30 days of the last dose occurred in 12% (19/158) of patients treated with COPIKTRA and in 4% (7/155) of patients treated with ofatumumab. Serious adverse reactions were reported in 73% (115/158) of patients treated with COPIKTRA and most often involved infection (38%; 60/158) and diarrhea or colitis (23%; 36/158). The most common adverse reactions with COPIKTRA (reported in ≥20% of patients) were diarrhea or colitis, neutropenia, pyrexia, upper respiratory tract infection, pneumonia, rash, fatigue, nausea, anemia, and cough.

For specific information on the management of the adverse reactions above, please review Dose Modifications for Adverse Reactions within the full Prescribing Information.

DRUG INTERACTIONS

CYP3A4 Inducers: Coadministration with a strong or moderate CYP3A4 inducer may reduce COPIKTRA efficacy. Avoid coadministration with strong or moderate CYP3A4 inducers.

CYP3A4 Inhibitors: Coadministration with a strong CYP3A4 inhibitor may increase the risk of COPIKTRA toxicities. Reduce COPIKTRA dose when co-administered with a strong CYP3A4 inhibitor.

CYP3A4 Substrates: Coadministration of COPIKTRA with sensitive CYP3A4 substrates may increase the risk of toxicities of these drugs. Consider reducing the dose of the sensitive CYP3A4 substrate and monitoring signs of toxicities of the co-administered sensitive CYP3A4 substrate.

Please see the Prescribing Information, including Boxed Warning.

To report Adverse Reactions during use of COPIKTRA, contact Secura Bio, call 1-844-9Secura or 1-844-973-2872 and/or to FDA at www.fda.gov/medwatch or call 1-800-FDA-1088.

(Press release, Secura Bio, NOV 4, 2026, View Source [SID1234674243])

On November 4, 2026 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, reported that new clinical data across the company’s rare hematology portfolio will be featured in oral and poster presentations during the 68th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH 2026) in New Orleans, Louisiana, December 12-15, 2026.

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"As Agios continues to expand its impact across rare hematologic diseases, ASH (Free ASH Whitepaper) 2026 represents a key opportunity to highlight the strength and differentiation of our portfolio," said Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D, Agios. "In sickle cell disease, new clinical data from the RISE UP Phase 3 trial show that mitapivat can deliver durable hemoglobin improvements for up to two years. RISE UP analyses also show the potential for mitapivat to improve measures of kidney function and cardiac remodeling in this population, which may reflect its unique dual activation of two pyruvate kinase isoforms – PKR expressed in red blood cells and PKM2 expressed in organ tissues. We will also present the first data from our AG-236 clinical program showing sustained, on-target activity that supports an every-six-month dosing schedule in polycythemia vera, potentially offering patients a best-in-class profile. Together, our ASH (Free ASH Whitepaper) 2026 data reinforce the potential for these medicines to transform the treatment of rare hematologic diseases with significant patient needs, and we look forward to sharing them with the hematology community at the congress next month."

Select Presentations at ASH (Free ASH Whitepaper) 2026
Mitapivat, oral pyruvate kinase (PK) activator

An oral presentation describing hemoglobin improvements with long-term mitapivat treatment (up to 115 weeks) among patients with sickle cell disease in the open-label extension (OLE) period of the RISE UP Phase 3 trial, during which all patients receive mitapivat. Of the 176 patients who completed the double-blind period of RISE UP, a total of 174 (118 from the mitapivat arm and 56 from the placebo arm) enrolled in the OLE period. Among all 118 patients continuing on mitapivat, 62.7% (n=74) achieved a hemoglobin improvement (defined as ≥1.0 g/dL increase from baseline in average hemoglobin during any two consecutive study visits) during the OLE period. Notably, of the 63 non-responders during the double-blind period, 39.7% (n=25) achieved a hemoglobin improvement with continued mitapivat treatment during the OLE period. Additionally, of the 56 patients who crossed over from placebo to mitapivat, 51.8% (n=29) achieved a hemoglobin improvement during the OLE period. The safety profile in the OLE period was consistent with that observed during the double-blind period. These results continue to support the potential of mitapivat as a durable, disease-modifying treatment for patients with sickle cell disease.
An oral presentation evaluating changes in kidney filtration rate, as measured by estimated glomerular filtration rate (eGFR), during the double-blind period of the RISE UP Phase 3 trial of mitapivat in sickle cell disease. In this condition, chronic hemolysis and anemia can cause the kidney to filter blood at an abnormally high rate (indicated by elevated eGFR), and this hyperfiltration is associated with long-term kidney damage. In a post hoc analysis, patients who received mitapivat had an early and sustained reduction in eGFR: among 132 patients with hyperfiltration (eGFR >135 mL/min/1.73 m²) at baseline, mitapivat-treated patients (n=92) showed a 14.7 mL/min/1.73 m² decrease in eGFR at Week 52, compared with a 4.9 mL/min/1.73 m² decrease with placebo (n=40). This reduction was observed regardless of whether patients achieved a hemoglobin response; taken together, these data suggest that the renal effects of mitapivat may be driven by both PKR- and PKM2-mediated mechanisms.
A poster presentation showing favorable cardiac remodeling among patients with sickle cell disease who achieved a hemoglobin response with mitapivat during the double-blind period of the RISE UP Phase 3 trial. This effect was evaluated by left ventricular mass index (LVMI) and left atrial volume index (LAVI), two measures of cardiac size and structure that can be abnormally elevated in sickle cell disease due to chronic hemolysis and anemia. This post hoc analysis found that, at Week 52, LVMI decreased from baseline by 5.9 g/m² among hemoglobin responders in the mitapivat arm (n=40), whereas it increased by 5.8 g/m² among non-responders (n=45) and by 4.6 g/m² in the placebo group (n=27). Hemoglobin responders also achieved a greater decrease from baseline in LAVI at Week 52 (4.7 mL/m²; n=35) compared with non-responders (1.7 mL/m²; n=41) and the placebo group (0.2 mL/m²; n=27). These data indicate that the anti-hemolytic effect of mitapivat may be a key driver of these improvements in cardiac remodeling, which, alongside preclinical evidence showing that PKM2 activation can attenuate cardiac fibrosis, suggests a potential dual cardiac benefit of mitapivat.
AG-236, small interfering RNA (siRNA) targeting TMPRSS6

A poster presentation highlighting first-in-human data from a Phase 1 single ascending dose study of AG-236 in 24 healthy volunteers. The study showed that AG-236 produced sustained increases in hepcidin – a key regulator of iron homeostasis – and decreases in circulating iron availability, including reductions in serum iron and transferrin saturation through Month 6 post-dose. Biomarkers of erythropoiesis (red cell production), hemoglobin and hematocrit, were also reduced through Month 6. AG-236 was well tolerated across all dose levels (1.0, 3.0, and 9.0 mg/kg), with no serious adverse events and no dose-limiting toxicities. In July 2026, Agios announced plans to advance AG-236 into a Phase 2/3 development program in polycythemia vera, with initiation of the Phase 2 portion expected in the second half of 2026.
In total, 14 abstracts led by Agios and external collaborators have been accepted for presentation and publication at ASH (Free ASH Whitepaper) 2026.

ASH 2026 Accepted Abstracts

Title Number Date/Time Presenter Acceptance
Sickle Cell Disease
Long-term hemoglobin
response from the Phase
3 RISE UP trial of
mitapivat in patients with
sickle cell disease 336 Saturday,
December 12, 2026,
5:15 – 5:30 p.m. CST Kevin Kuo, M.D.,
University of Toronto Oral
Mitapivat is associated
with early and sustained
improvements in eGFR
among patients with
sickle cell disease: A post
hoc analysis of the Phase
3 RISE UP trial 741 Monday, December
14, 2026, 11:00 –
11:15 a.m. CST Kenneth Ataga, M.D.,
Center for Sickle Cell
Disease at University of
Tennessee Health
Science Center Oral
Mitapivat is associated
with favorable cardiac
remodeling in
hemoglobin responders
with sickle cell disease: A
post hoc analysis of the
Phase 3 RISE UP trial 1158 Saturday,
December 12, 2026,
5:30 – 7:30 p.m. CST David Tofovic, M.D.,
University of Illinois
Hospital Poster
Mitapivat rapidly restores
red blood cell membrane
integrity: A window for
pre-emptive intervention
in sickle cell vaso-
occlusive crisis 1149 Saturday,
December 12, 2026,
5:30 – 7:30 p.m. CST Swee Lay Thein, MBBS,
DSc, FRCP, FRCPath,
MRCP, MRCPath,
National Heart, Lung &
Blood Institute Poster
Ongoing burden of sickle
cell disease among
individuals treated with
newly approved
disease-modifying
therapies: A real-world
claims analysis using
United States Medicaid
data 4434 Sunday, December
13, 2026, 6:00 – 8:00
p.m. CST Nirmish Shah, M.D., Duke
University Adult
Comprehensive Sickle
Cell Center Poster
Longitudinal trends in
renal biomarkers of US
patients with sickle cell
nephropathy 4712 Monday, December
14, 2026, 6:00 – 8:00
p.m. CST Santosh Saraf, M.D.,
University of Illinois
Hospital Poster
Impact of patient-
reported pain crises on
the lives of adults with
sickle cell disease in the
United States: Results
from the real-world RAISE
study 6194 Monday, December
14, 2026, 6:00 – 8:00
p.m. CST Sherif Badawy, M.D.,
Northwestern University
Feinberg School of
Medicine Poster
Trial in progress: Study
design of the
randomized, double-
blind, Phase 3 REIGNITE
trial to evaluate the effect
of mitapivat versus
placebo on transfusion
burden in patients with
sickle cell disease 4686 Monday, December
14, 2026, 6:00 – 8:00
p.m. CST Julia Xu, M.D., MScGH,
University of Pittsburgh
Medical Center Poster
Mitapivat promotes
terminal erythroid
maturation in patient-
derived sickle cell
disease erythroid
cultures 4668 Monday, December
14, 2026, 6:00 – 8:00
p.m. CST Dr. Wassim El Nemer,
Ph.D.,
French National Blood
Service (EFS)
Poster
Relationship between
markers of hemolysis and
clinical outcomes in
sickle cell disease: A
systematic literature
review and meta-analysis N/A N/A Kenneth Ataga, M.D.,
Center for Sickle Cell
Disease at University of
Tennessee Health
Science Center
Publication
Thalassemia
Long-term changes in
iron overload and
markers of iron regulation
and erythropoiesis with
mitapivat in patients with
α– or β–non–transfusion-
dependent thalassemia:
Results from the Phase
3 ENERGIZE open-label
extension 9 Saturday,
December 12, 2026,
10:00 – 10:15 a.m.
CST Hanny Al-Samkari, M.D.,
Mass General Brigham
Cancer Institute Oral
Clinical and patient-
centered implications of
reduced transfusion
burden with mitapivat in
transfusion-dependent
thalassemia: A post hoc
analysis of ENERGIZE-T 4655 Monday, December
14, 2026, 6:00 – 8:00
p.m. CST Sujit Sheth, Weill Cornell
Medicine Poster
Polycythemia Vera
Safety and tolerability of
AG-236, an
investigational TMPRSS6-
targeting small interfering
RNA for polycythemia
vera, in healthy
participants 3772 Sunday, December
13, 2026, 6:00 – 8:00
p.m. CST
Jonathan Meiman, M.D.,
University of Wisconsin-
Madison Poster
Other Conditions
Distinct metabolic
signatures differentiate
Rps19 and Rpl5
haploinsufficiency in
Diamond-Blackfan
Anemia syndromes 3183 Sunday, December
13, 2026, 6:00 – 8:00
p.m. CST Jonathan de Wilde, M.D.,
University Medical Center
Utrecht, Netherlands
Poster

Please refer to the ASH (Free ASH Whitepaper) 2026 website for full session details and data presentation listings and visit the Agios booth (#929) onsite.

ASH 2026 Investor Event
Agios will host an ASH (Free ASH Whitepaper) 2026 virtual investor event on Monday, December 14, at 1:00 p.m. EST (12:00 p.m. CST). The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the "Events & Presentations" tab. A replay of the webcast will be available on the company’s website approximately two hours after the event.

About Mitapivat in Sickle Cell Disease
Sickle cell disease is a rare, inherited blood disorder in which abnormal hemoglobin causes red blood cells to become rigid and sickle-shaped, leading to chronic hemolysis (red blood cell destruction) that contributes to severe anemia and progressive, life-threatening complications, including damage to the lungs, kidneys, and heart. Mitapivat is an oral medicine designed to enhance red blood cell energy by activating the pyruvate kinase (PK) enzyme, which can increase levels of adenosine triphosphate (ATP) and lower levels of 2,3-diphosphoglycerate (2,3-DPG). Mitapivat has a unique dual mechanism of action that activates two isoforms of the PK enzyme – PKR expressed in red blood cells and PKM2 expressed in organ tissues – and has shown the potential to address both chronic hemolysis and pathways involved with long-term organ damage.

In July 2026, Agios announced that the U.S. Food and Drug Administration (FDA) accepted its supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease with a Priority Review. In October 2026, Agios announced the FDA extended the Prescription Drug User Fee Act (PDUFA) goal date for this sNDA by three months, from November 1, 2026, to February 1, 2027. The mitapivat sNDA is based on data from the global, randomized, double-blind, placebo-controlled RISE UP Phase 2 and Phase 3 trials (NCT05031780) in patients aged 16 years or older with sickle cell disease.

About AG-236 in Polycythemia Vera
Polycythemia vera (PV) is a rare blood cancer characterized by the overproduction of blood cells driven by an underlying genetic mutation in Janus kinase 2 (JAK2). Patients with PV typically have reduced levels of hepcidin, a critical hormone that regulates iron in the body. AG-236 is an investigational, small interfering RNA (siRNA) designed to silence the expression of the transmembrane serine protease 6 (TMPRSS6) gene, which normally downregulates hepcidin. By silencing TMPRSS6, AG-236 is designed to upregulate hepcidin, thereby restricting iron availability and controlling the overproduction of red blood cells associated with PV.

Based on promising results in a Phase 1 trial in healthy volunteers, Agios is advancing AG-236 into a Phase 2/3 development program (NCT07859423) in polycythemia vera, with initiation of the Phase 2 portion expected in the second half of 2026.

AG-236 is licensed from Alnylam Pharmaceuticals, Inc.

(Press release, Agios Pharmaceuticals, NOV 4, 2026, View Source [SID1234674242])

On November 4, 2026 CRISPR Therapeutics (Nasdaq: CRSP) reported that clinical data in patients with relapsed or refractory large B-cell lymphoma from the ongoing Phase 1/2 trial of zugocabtagene geleucel (zugo-cel), its investigational allogeneic CRISPR/Cas9 gene-edited chimeric antigen receptor (CAR) T cell therapy targeting CD19, will be presented in an oral presentation at the 68th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place December 12 to 15, 2026.

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Abstract Title: Zugocabtagene geleucel (zugo-cel), an off-the-shelf CRISPR-Cas9 engineered CD19 CAR T cell therapy demonstrates dose-dependent efficacy in highly refractory large B-cell lymphoma
Abstract Number: 706
Session Type: Oral Presentation
Session Title: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Next-Generation CAR T-Cell Therapy in Lymphoma: In Vivo Engineering and Beyond
Session Date and Time: Sunday, December 13, 2026, 5:15 p.m. CST

A copy of the presentation will be available at www.crisprtx.com once the presentation concludes.

About Zugocabtagene Geleucel (zugo-cel; formerly CTX112)
Zugo-cel is a wholly-owned, allogeneic chimeric antigen receptor (CAR) T cell therapy product candidate targeting Cluster of Differentiation 19 (CD19), in development for both autoimmune and immuno-oncology indications. The off-the-shelf therapy leverages CRISPR/Cas9 for targeted gene knockout and CAR insertion, enabling immune evasion and enhanced T effector cell potency, and is administered following a standard lymphodepletion regimen without the need for human leukocyte antigen (HLA) matching. In autoimmune disease, zugo-cel is being investigated in three ongoing basket trials. The rheumatology basket trial is enrolling adult patients with systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis (IM). The hematology basket trial is enrolling patients with immune thrombocytopenic purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA). The neuroimmune basket trial is enrolling patients with progressive multiple sclerosis (PMS), neuromyelitis optica spectrum disorder (NMOSD), myelin oligodendrocyte glycoprotein antibody-associated Disease (MOGAD), N-methyl-D-aspartate receptor (NMDAR) and leucine-rich glioma-inactivated Protein 1 (LGI1) autoimmune encephalitis (AIE), and stiff person syndrome (SPS). In immuno-oncology, zugo-cel is being investigated in a trial in adult patients with relapsed or refractory B-cell malignancies.

(Press release, CRISPR Therapeutics, NOV 4, 2026, View Source [SID1234674241])

On November 4, 2026 Solu Therapeutics, a biotechnology company pioneering novel therapies to eliminate disease-driving cells in cancer and immunology, reported that initial results from its first-in-human Phase 1 study of STX-0712 in patients with chronic myelomonocytic leukemia (CMML), and new preclinical data on STX-0640 have been selected for oral presentations at the 68th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held in New Orleans, LA on December 12-15, 2026.

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"We are excited to present the first clinical data from the ongoing Phase 1 of STX-0712 in patients with CMML, and new preclinical data on STX-0640, our potential first-in-class MRGPRX2-targeting mast cell depletor, at the upcoming ASH (Free ASH Whitepaper) Annual Meeting. Having two abstracts selected for oral presentation at the premier hematology meeting underscores the growing recognition of our differentiated approach to selectively eliminate disease-driving cells," said Philip Vickers, President and CEO of Solu Therapeutics. "With our Phase 1 study of STX-0712 ongoing in CMML, and recently expanded to include patients with AML, we continue to make meaningful progress in advancing this novel therapeutic approach in diseases with significant unmet need. We look forward to sharing the data and highlighting the breadth and potential of our pipeline."

Oral Presentation Details

Title: Initial results from a first-in-human Phase 1 study of STX-0712, a CCR2+ cytotoxicity targeting chimera (CyTAC) in patients with chronic myelomonocytic leukemia (CMML)
Session: Myelodysplastic Syndromes: Clinical and Epidemiological: Novel Targeted Agents and Combination Regimens in CMML, MDS, and MDS/MPN Overlap Neoplasms: Emerging Clinical Trial Data
Publication number: 532
Date & time: December 13, 2026 at 9:30 AM CT
Presenter: Guillermo Montalban-Bravo, MD; Associate Professor, Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas

Title: Preclinical evaluation of STX-0640, a first-in-class MRGPRX2-targeting mast cell depletor for systemic mastocytosis and related mast cell disorders
Session: Myeloproliferative Syndromes and Chronic Myeloid Leukemia: Basic and Translational: Triple S: MPN Goes Spatial, Selective and to the Cell Surface!
Publication number: 677
Date & time: December 13, 2026 at 5:30 PM CT
Presenter: Brandon Turunen, PhD; Chief Scientific Officer and Co-Founder, Solu Therapeutics

About STX-0712
STX-0712 is an investigational Cytotoxicity Targeting Chimera (CyTAC) targeting the G Protein-Coupled Receptor CCR2, a selective marker expressed at high levels on malignant monocytes and bone marrow blasts, which are key drivers of disease in chronic myelomonocytic leukemia (CMML), acute myeloid leukemia (AML), and other hematologic cancers. Designed to selectively eliminate CCR2-positive cells, STX-0712 has the potential to offer a more targeted and effective treatment option with minimal effects on non-malignant cells.

STX-0712 has received Fast Track Designation from the U.S. Food and Drug Administration and is currently being evaluated in a Phase 1 study in patients with relapsed/refractory CMML and AML.

About STX-0640
STX-0640 is an investigational, potential first-in-class mast cell depletor and development candidate for systemic mastocytosis and immuno-dermatologic diseases. Built on the CyTAC platform, STX-0640 combines a selective small-molecule binder of MRGPRX2, a receptor selectively expressed on disease-driving tissue-resident mast cells that is poorly accessible to conventional antibodies, with a T cell engager. Designed to eliminate mast cells rather than merely inhibiting their activity, STX-0640 has the potential to enable deeper and more durable clinical responses.

(Press release, Solu Therapeutics, NOV 4, 2026, View Source [SID1234674240])